Using targeted DNA sequences to increase protein production for treating neurodegenerative diseases
Official title: Boosting protein synthesis using antisense oligonucleotides to treat neurodegeneration
Funded at Washington University · NIH-11105763 · Data updated 2026-11-12
This study is looking at a new way to help older adults with neurodegenerative diseases, like ALS and frontotemporal dementia, by using a special treatment to boost the production of important proteins in the brain that are lost as we age.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | June 1, 2024 |
| Project end | November 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $53,312 |
| NIH project number | 5F30AG082394-02 |
| Project ID | NIH-11105763 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include older adults or individuals diagnosed with neurodegenerative diseases such as amyotrophic lateral sclerosis or frontotemporal dementia.
✕Less likely to help
Patients with neurodegenerative conditions not related to protein synthesis or those who are not of advanced age may not benefit from this research.
What this research studies
This research focuses on addressing the loss of essential proteins in the aging brain, which contributes to neurodegenerative diseases like amyotrophic lateral sclerosis and frontotemporal dementia. The approach involves using antisense oligonucleotides (ASOs) to enhance protein synthesis by blocking specific RNA elements that inhibit translation. By improving the stability and translation efficiency of messenger RNA (mRNA), the goal is to restore the production of protective proteins that are diminished with age or disease. Patients may benefit from a novel therapeutic strategy that aims to counteract the effects of neurodegeneration.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that enhance protein production in the brain, potentially slowing or reversing neurodegenerative processes.
How similar studies have gone: While the use of antisense oligonucleotides is a relatively novel approach in this context, similar strategies have shown promise in other areas of genetic and neurological research.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Boros, Ben David — Washington University
- Study coordinator: Boros, Ben David
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.